Arthrogryposis multiplex congenita (AMC) is a group of disorders defined by two or more contractures in different body areas; while genes encoding sarcomeric proteins are usually involved in its pathogenesis, the role of the dystrophin complex is not well studied in AMC. Utrophin, encoded by the UTRN gene, is an important fetal dystrophin homologue and was the focus of a recently presented study.
Applied Biologics LLC has initiated a preclinical research program focused on osteoarthritis and other degenerative diseases. The program marks an expansion of the company’s research and development efforts beyond chronic wounds.
Edgewise Therapeutics Inc. is pulling in $1.55 billion up front by selling its muscular dystrophy business, including its fast skeletal myosin inhibitor, sevasemten (EDG-5506), to Servier SA. The deal is potentially worth up to $2.65 billion when including a potential $1.1 billion in milestone payments.